A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

Factor XIII Deficiency Clinical Trial

Official title:

A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00883090
• Other study ID #: BI71023_2002
• Secondary ID: 2009-010387-4114
• Status: Completed
• Phase: Phase 2
• First received: April 16, 2009
• Last updated: December 7, 2011
• Start date: May 2009
• Est. completion date: April 2010

Study information

• Verified date: December 2011
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationSpain: Comité Ético de Investigación Clínica
• Study type: Interventional

Clinical Trial Summary

Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: April 2010
• Est. primary completion date: April 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Written informed consent/assent for study participation obtained before undergoing any study-specific procedures

• Documented congenital FXIII deficiency that requires prophylactic treatment with a FXIII containing product.

• Males and females of any age with congenital FXIII deficiency.

• Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive

Exclusion Criteria:

• Diagnosis of acquired FXIII deficiency

• Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0

• Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency

• Known or suspected to have antibodies towards FXIII

• Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)

• Positive result at screening for human immunodeficiency virus (HIV)

• Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal

• Fibrinogen < lower limit of normal

• Active bleeding

• Pregnant or breast-feeding

• Intention to become pregnant during the course of the study

• Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study

• Surgical procedure anticipated during the study period

• Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance

Study Design

Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Factor XIII Deficiency

Intervention

Biological:
• FXIII Concentrate (Human)
Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.

Locations

Country	Name	City	State
Spain	Study Site	Santa Cruz de Tenerife
United States	Study Site	Boston	Massachusetts
United States	Study Site	Dothan	Alabama
United States	Study Site	Orange	California
United States	Study Site	San Francisco	California
United States	Study Site	Stockton	California

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

United States,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Peak FXIII Concentration at Steady State		12 weeks	No
Primary	Trough FXIII Concentration at Steady State		12 weeks	No
Primary	Time to Peak Concentration		12 weeks	No
Primary	Incremental Recovery	Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.	12 weeks	No
Primary	Terminal Half-life		12 weeks	No
Primary	Area Under the Curve at Steady State		12 weeks	No
Primary	Clearance		12 weeks	No
Primary	Volume of Distribution at Steady State		12 weeks	No
Primary	Mean Residence Time		12 weeks	No
Secondary	Adverse Events	Number of participants with an adverse event	16 weeks	Yes
Secondary	Laboratory Safety Parameters	Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.	16 weeks	Yes
Secondary	Vital Signs	Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.	16 weeks	Yes

External Links

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